Researchers at Children’s Hospital of Philadelphia (CHOP) recently reviewed best practices, guidelines, and care path objectives for early-onset sepsis (EOS), an invasive bacterial or fungal infection that occurs in infants within the first week of life and can be fatal. Their recommendations were recently published in Clinics in Perinatology.

An interprofessional team at Children’s Hospital of Philadelphia is transforming CHOP’s approach to fetal medicine through a first-of-its-kind data integration platform. The Clinical Outcomes Data Archive (CODA) platform offers clinicians real-time data to help inform earlier interventions and treatment strategies in CHOP’s Richard D. Wood Jr. Center for Fetal Diagnosis and Treatment (CFDT). A paper outlining CODA’s benefits, applications and potential broader impact was recently published in the journal Fetal Diagnosis and Therapy.

Researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania unlocked important new insights in how the immune system selects the right signals to alert T cells, a vital step in the prevention and treatment of serious illness. The findings were published in the journal Proceedings of the National Academy of Sciences (PNAS).

Researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania developed a new screening technology, Aptamer-based T Lymphocyte Activity Screening and SEQuencing (ATLAS-seq), to better identify antigen-reactive T cells that are more likely to offer greater immune responses against cancer cells.

Researchers at Children’s Hospital of Philadelphia today announced results from the first ever international survey of trochleoplasty experts, which they hope will help guide current practice and future research regarding this procedure.

Researchers from the Children’s Hospital of Philadelphia (CHOP) in collaboration with the School of Veterinary Medicine at the University of Pennsylvania, Perelman School of Medicine at the University of Pennsylvania and Vanderbilt University Medical Center found that venous endothelial cells (VECs), a specific type of lung blood vessel cell, can help fix damaged blood vessels in the lungs following lung injury or disease. Their findings were recently published in Nature Cardiovascular Research.

Researchers at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a first of its kind gene therapy model that offers a potential breakthrough in treating X-linked sideroblastic anemia (XLSA), a rare congenital anemia caused by mutations in the ALAS2 gene crucial for the synthesis of heme, a key compound in hemoglobin. This study marks the first time researchers studied gene therapy to treat this disease, which the authors underscore could have an impact on a broad spectrum of diseases. The research was published today and featured on the cover of the journal Blood.

Researchers at Children’s Hospital of Philadelphia (CHOP) and the Children’s Oncology Group (COG) found that larotrectinib, an oral drug that stops cancer-cell growth, was highly effective in pediatric patients with newly diagnosed infantile fibrosarcoma (IFS) or other solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation. The study, published today in the Journal of Clinical Oncology, is the first COG trial to test precision medicine in a front-line setting across all different types of solid tumors based on a genetic biomarker rather than histology. It is poised to redefine the approach to treatment for newly diagnosed fibrosarcoma and other solid tumors with the NTRK gene fusion, potentially reducing or eliminating the need for these patients to receive chemotherapy.

Researchers at Children’s Hospital of Philadelphia (CHOP) and the Children’s Oncology Group (COG) announced the results of a Phase 3 study that demonstrated adding the bi-specific T-cell engager, blinatumomab, to chemotherapy for newly diagnosed National Cancer Institute (NCI) standard risk (SR) B-cell acute lymphoblastic leukemia (B-ALL) pediatric patients significantly improves survival outcomes. The results were published today in the New England Journal of Medicine and will be presented at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition on December 8.

Study identified a drug for potential use in future precision medicine clinical trials